Committee on Public Health Ordinary meeting
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SANT European Parliament
Follow its sittingsJessica Polfjärd
Thank you, chair. And I'd like to begin by thanking the rapporteur and her team for the draft report and for the constructive start of these negotiations. And like you rightly pointed out, cardiovascular health is important and often underdiscussed. So therefore, I think it's very good that we have this discussion, and we also have the possibility to write this report. As it is the leading cause of death in Europe, the fight against cardiovascular disease is an essential part of making the EU healthier and stronger.
And I'm happy to see that many colleagues in this committee have been actively engaging with this file, and that there have been many amendments tabled, not least from the EPP. And as I mentioned at the previous time we discussed this topic, it is clear that there is a broad agreement on the need to strengthen prevention. Prevention of disease should always be a priority. And prevention means encouraging physical activity, promoting health awareness and healthier diets, not least the scientifically supported Nordic and Mediterranean diets. Prevention also means considering the interaction between other conditions and cardiovascular health, such as diabetes, obesity, and respiratory illness.
How we tackle these will be important. For example, vaccination against influenza is one tool that we also have to take into consideration. The role of the treatment and the importance of integrated pathways to care is another area where I want to highlight this issue.
And in this regard, research and development of new treatments and the importance of Europe's cutting-edge life science sector must also be considered and recognized. Mine and EPP's amendments aim at putting more focus on these areas while having due respect for the principle of subsidiarity and allowing member states to create strategies that
suit their national environments and the member states the best. Cardiovascular disease affects all of society, and the response must also include all of society, including the EU, national government, public and private sector institutions as well as patients and civil society. Again, I want to thank the rapporteur and her team and the other shadows for the work so far. And I look very much forward to continuing the work with the rapporteur and with the other groups to create a report that ensures that all of Europe joins the fight against cardiovascular disease.
Speaker 10
Honorable members here, chair. First of all, I'd like to thank the rapporteur, Romano, and the shadows for their work, and also for their technical assistance for this work. And a high number of amendments show that cardiovascular diseases are of extreme importance. These diseases continue to be the primary cause of death in Europe. Almost 2 million people die every year in Europe.
Speaker 9
We need a multilevel approach, therefore, based on prevention, early detection, and rehabilitation. I'm glad that the draft report takes these into account, and I welcome that it emphasizes the protection of the youth. Our amendments intend to strengthen this approach. I'm convinced that if we focus on all the three levels like healthy life, screening, and rehabilitation, we are able to provide real added value. Moreover, I think we need balanced compromises, especially in those questions, for instance, in the assessment of certain risk factors where the technical level could not reach an agreement.
The report should stay within the remit of the Sandd Committee, taking into account that health care is primarily a member state competence, especially tax policy falls into the remit of the member state. Therefore, we have to show restraint in these areas. Thank you again for your work, and I look forward to continuing it.
Vlad Vasile-Voiculescu
I will read on behalf of my colleague, MEP Veronika Ostikranova. Her text. So dear colleagues, I would like to highlight several priorities that are particularly important for my group and that I will be working on, to be implemented in the reports. First, we must significantly strengthen prevention, both primary and secondary. Today, harmful products such as alcohol and colorful or flavored vapes are marketed online in a very appealing way, especially to children.
These strategies normalize harmful behaviors and expose children and young people to lifelong cardiovascular risks. Prevention from a young age must, therefore, be central to our approach. Stronger school-based programs and stricter limits on the marketing of unhealthy and addictive products are essential. In this context, the upcoming evaluation of the other visual media services directive offers a key opportunity to better protect young people. Age verification can also play an important role.
The EU digital identity wallets, as expected to be implemented by the end of this year, could help prevent children from purchasing restricted goods, such as tobacco or alcohol. At the same time, we should work towards harmonized regulation of marketing across the digital environment, including social media platforms, websites, gaming platforms, and apps. However, prevention also means ensuring regular health checks, so that people truly know their numbers. And when cardiovascular diseases with a genetic predisposition are detected, family screening should be provided. I would also like to stress the importance of a patient centered approach.
Young people living with cardiovascular disease often have very different needs from older patients when it comes to rehabilitation, for instance. Personalized care can significantly improve their mental well-being and overall quality of life. Finally, addressing gender disparities in cardiovascular health is crucial. Women still face barriers in prevention, diagnosis, and treatment. We need balanced gender representation in clinical research and gender-sensitive analysis of outcomes to improve care for everyone.
Thank.
Martin Häusling
Thank you very much for the excellent report, and we've obviously played our role in that. I think the first thing we need to make clear is the importance that cardiovascular disease has for Europe, almost 2 million deaths a year. That's already been mentioned, 280 million for follow-up costs. And therefore, this is an appeal to all to be more committed to this topic. There's a very different approach taken in different member states.
A lot of the disease is avoidable. The risk factors such as tobacco, alcohol, poor diet, and environmental impacts are well known. Now there are some things we can do in Europe in order to reduce that impact. The environment, for example, is an area where we could clean up the air. We need to do more about that.
Chemicals is an area. PFAS is one that we need to think about. Some groups are particularly exposed because of their profession. And then the access to healthy food. We've got an intensive debate going on in Germany about the provision of healthy food, particularly for children.
And I think this is a preventive approach we need to adopt, need better labeling of foodstuffs on the packaging. And we need to have those foodstuffs with high levels of sugar, fat, and salt properly labeled. There are some tax measures that can be deployed in order to help people make the right choices for sustainable and healthy food. These need to be made mandatory, and they need to be made visible. And we need also to ensure that the One Health approach is adopted for micro, sorry, for bacterial resistance.
Tobacco and alcohol, I've already mentioned them. We do really need to think about how we can strengthen the restrictions on tobacco consumption and alcohol consumption. I think the primary approach would be by making it more expensive. So there are measures of a preventive kind as well that can be adopted, and we need to have awareness raising amongst children. Now we need to think about whether you can act and should act.
And it's clear that we shouldn't be appealing only to individual action, but to early diagnosis, regular monitoring, coordination, and the early recognition of key risk factors. We're looking forward to the report, and we will be playing as a group a constructive role in that. I'd also like to say a big thank you to the rapporteur and the colleagues who have worked on this. We're talking, after all, about a very important topic. Cardiovascular disease is one of the biggest causes of death, but it's about 80 percent avoidable.
Sebastian Everding
So prevention ought to be at the top of the list when it comes to what we're doing in the EU. In our group, we see an important approach to food. If people are consuming red meat too much, maybe we could move to plant-based foods that help to prevent these diseases. And therefore, the promotion of plant-based foods is not just an ideological matter. It's based on solid science.
But at the same time, it's all the more worrying that the right leading groups on nicotine, alcohol, energy groups are trying to make them appear harmless, and saying that there should be protections for industry. That's extremely worrying. And yet the scientific evidence is perfectly clear. These things damage the health of the citizens of the EU. Prevention, this is very important.
Requires children to find out about this to be educated about, and this needs to be happening across the board in different geographies. It often depends on whether you grow up in the countryside or in a city as to whether you have this information, so it needs to be made available to all. And then sex-specific aspects are important, shouldn't be ignored. Women are often diagnosed too late when it comes to cardiovascular disease, and there's late, inadequate treatment. And then stress, air pollution, let's not leave noise out of it.
All of these play an important role in cardiovascular disease development. So the facts are on the table, colleagues. If anybody is hesitating, then not looking at these facts, and this is a risk to all of our health.
The Nevada del Carmen, please.
Elena Nevado del Campo
Very much, chair. I'd like to thank all of the work that's been done by the rapporteur as well as the shadow rapporteurs. More than 1,000 amendments, that's a lot of amendments, and it shows how important this topic is and how much work has been doing, has been happening on cardiovascular diseases. I would also like to say that many of the amendments affect some whole areas in Spain. That's one of the provinces that I'm from, because you can also die from other things.
You can die of hunger, and often there's a lot of hypocrisy within governments. Now the life expectancy in Spain is the highest in the European Union. That has a lot to do with the Mediterranean diet, our lifestyle. And so we don't, well, there's a lot of alarmist discourse around wine. I mean, we're not going to go back to those old narratives, but we need to talk about moderation and responsible consumption.
And so I would encourage everyone to continue working in that direction, again, highlighting that the first cause of death of women is breast cancer, and we would like to have the best report possible with consensus, bearing in mind all of these specific issues. Thank you very much, Chair. First, wanted to congratulate the rapporteur on her work and to wish her good luck with these thousands of amendments. It will be a lot of work. Two comments.
Christophe Clergeau
First on taxation. I heard what our PFE colleagues said who they don't want a European tax, but Commissioner Varelli was the one who suggested a junk food tax to have revenue to feed into the European budget on public health. And that seemed to be a good idea. I think that should be an open debate. And then the second comment, which is most important, is on prevention.
This is going to be essential to really make it clear what we need in terms of prevention. I think we can all agree that we need better detection, early detection, and early diagnostics. We need to look at individual behavior patterns, whether that's diet or smoking, etc. But we wouldn't want to only forget about all of the other things and only look at individual issues. We can look at the diet, for example.
That perhaps comes more under envy than our committee, but we need to be clear about what we want to look at in a European Union when we're talking about diet, labeling, ultra processed food. But we do have to bear in mind the social determinants and the environmental determinants of this. Because in the past, we have rightly highlighted tobacco, etc., because we had information. But now we have studies that show the impacts of poverty, quality of life, etc. Tomorrow, we're going to have a presentation on the follow-up of the report on the exposome.
But all of the things that we're exposed to and how they lead to chronic diseases, whether that's cancer, cardiovascular diseases, degenerative diseases, all of these are things that we have to bear in mind in our discussions. There is a collective social and environmental dimension of cardiovascular diseases.
Margarita Della Pizzacarion, please.
Thank.
Speaker 20
Thank you very much, chair. Ladies and gentlemen, I first wanted to start thanking the shadow reporters and the reporters. I think this report clearly shows that amendments are an opportunity for us to all work together and share our ideas, so that this strategy is as enriching as possible, and all political groups are able to work together. I think this cardiovascular strategy shows that we can all share our experiences from our own country. Now just to complement, when we're talking about cardiovascular diseases, we're referring to a series of infections which affect the heart and cardiovascular system, and have, and can have an impact on all of the organs in our body.
With our amendments, we wanted to highlight the impact of strokes, which can have an impact on the motor system and cognitive system. Now being able to detect the symptoms is essential so that you can deal with it if there is an emergency. There also need to be tangible actions to be able to recognize what's happening, as well as having more other measures like more defibrillators in public spaces. There needs to be education for the public so they know how to act if someone is suffering from a heart attack. Also wanted to highlight that we need to talk about the important role of pharmacies or pharmacists and medical staff.
Speaker 21
We need to improve their training when it comes to helping patients, and their involvement can help detect any symptoms and ensure that patients are respecting their treatments. Thank you very much.
Adam Jarubas
Thank you, colleagues, for your interventions. Now I give the floor to the representative of the commission, Marianne Taki, Head of the Disease Prevention and Health Promotion Unit at DG Sante, for her reaction. Two minutes, please.
Speaker 22
Dear honorable members, good morning. Thank you for the importance you are giving to addressing the challenges of cardiovascular diseases in the EU. I have taken note of the points raised at today's meeting. On December 16, we adopted the first ever EU cardiovascular health plan, the Safe Hearts Plan, that was developed in consultation with member states and stakeholders. It is an ambitious approach to improve people's lives at the personal level and at the societal level.
The commission is now focusing on the implementation. Through the EU Cares for Your Heart program, we will provide tailored support to member states in developing and implementing national stand-alone or integrated cardiovascular health plans by 2027. This programme will build on joint actions to prevent NCD and Jakarti. Also, through our work with international partners such as the World Health Organization and OECD, we will support member states in strengthening prevention through capacity building and health literacy activities, in particular among vulnerable groups. Among our priorities this year is a proposal for a council recommendation on health checks for cardiovascular diseases.
It aims to help member states strengthen and align their efforts on early detection and screening. We will also launch an awareness campaign, Know Your Numbers, in support of this proposal. We are currently working very closely with the member states on the development of the council recommendation, and it will be on the evidence collected by the OECD. The specific needs of vulnerable groups, including young people, women, and socioeconomically disadvantaged groups, will be considered. The plan also aims to improve the quality and consistency of personalized and integrated care pathways through a proposal for a Council recommendation intended to be adopted in 2027.
Another objective is to foster innovation and integration of AI and digital technologies for early detection, personalized prevention, and integrated care through a EUR 20,000,000 project under the EU for Health. Finally, the SafeHEARS plan aims to address health inequalities through the development of an EU cardiovascular health inequalities dashboard. We look forward to continued exchanges with the European Parliament. Thank you.
Adam Jarubas
Thank you. Finally, I would like to ask the reporter for her reaction to the comments made by MEPs. Romana, are you there?
Speaker 3
Yes. I'm here. Thank you, chair. Thank you all once again for your contribution and for your hard work on this report and the amendments. As I said at the beginning of our discussion, it was expected, you know, that there will be some points on which we would not fully agree.
However, today's discussion, today's exchange has been, I would say, very constructive and has helped us better understand each other's perspective. To my colleague, I could not agree more. The prevention requires a whole of society approach. To colleague, indeed, do need to respect the competencies. And regarding taxation, we have great examples in how EU guys did the back taxation with respect to the competencies of the member states.
Colleague, I agree we need to protect the young people from the marketing of harmful industries. Housing and everything, food and environments play an important role in cardiovascular health, and that's why I dedicated a section in the report to it. Christophe, thank you for your continued support and interest in this file. And, my colleague, Della Pisa Carreon, I agree that both community and the hospital pharmacist play an important role, too. So, finally, I would just make a comment that this report comes at a very important moment.
We have a, I would say, once in a decade opportunity to strengthen the EU approach to cardiovascular health. And for that reason, I believe it is important that we continue to work in a constructive spirit as we move forward in the process. Some differences remain. Yes. It's inevitable, but I'm confident that, through continued dialogue, we can reach a strong and balanced outcome, one that reflects the ambition needed to address this major public health challenge.
Thank you all, and thank you, Chair.
Yes. Thank you. Thank you.
Short interventions.
Speaker 23
Yeah. Yeah. Very short. Very short. Just a few comments.
First, I do not believe that this is any place for ideology in this report because this is a matter of public health, and our policy choices should be guided by the best available scientific evidence. That is why I find it difficult to accept an approach that places red meat in the same category as junk food and ultra-processed foods without making the necessary distinctions. High quality unprocessed red meat can be part of a balanced diet and is an important source of protein and other nutritions. It is precisely this kind of dogmatic and undifferentiated thinking that prevents us from examining cardiovascular health in an objective and evidence-based way. The reality is not all products present the same level of risk.
Risk depends on the nature of the product, its composition, its level of processing, and the pattern of consumption. Processed red meat does not present the same risk profile as lean, processed red meat. And in the same way, different alcoholic products have different patterns of harm, and different tobacco and nicotine products also differ in their health impact. Recognizing these differences is not ideological. It is simply a matter of scientific honesty and sound policymaking.
For that reason, I welcome the inclusion of the principle of differentiating products according to the level of risk they pose to cardiovascular health. That is the right approach, proportionate, evidence based, incredible. I also welcome the emphasis on protecting vulnerable groups, young people, and children. We know that some products are more harmful than others. Our duty is therefore clear: to act more strongly where the risk is highest and to ensure that those who are most vulnerable are properly protected.
And my last comment for today, which I want to emphasize, concerns alcohol. We need to inform the consumer fairly. Far too long have we protected and promoted alcohol consumption in Europe in the name of culture and habit. We finally need to change this approach. Thank you.
Adam Jarubas
Thank you. Let's now move to the next point, establishing the European Competitiveness Fund, including the specific program for defense research and innovation activities, consideration of amendments. We start with the consideration of amendment on the Sandd opinion to the European Competitiveness Fund, and the lead committee is ITRE. I will give the floor first to the Sandd rapporteur, then to the Sandd shuttles, and to any members who ask for the floor, best based on catch the eye. We will finally give the floor to the commission.
If you want to take the floor, just raise your hands. Mister Kanev, rapporteur for the Sand Opinion, you have the floor for 4 minutes, please.
Speaker 25
Thank you very much, chair. I guess it won't take 4 minutes, so we can save some time. Because, I would first like to thank all the shadows and all the members of the committee who have filed already amendments. And, I must consider, there is a very strong spirit of cooperation among the different groups, the shadow team and the maps. So we're really looking in the same direction, with our amendments.
Speaker 24
First, I must underline there is a broad agreement that we need earmarking of the funds dedicated to health. All the political groups, one way or another, have proposed that the health funds are clearly dedicated to health policy. And, I would add that, furthermore, we need much more clarity whether the ECF health budget is still dedicated to health policy proper or health related industrial policy. That is a very big difference. If we look at the objectives of ECF as a whole, we must consider that health related industrial policy much closer to the goals of the whole legislation, of the whole regulation.
But under the MFF, there is no other health budget window. So if we want to maintain health as a main priority of the European budget, we need to clearly dedicate funds to health policy, including prevention, treatment, etcetera. And since European level health policy is one of the main priorities of the EPP for a second term now. So my position as a rapporteur is clearly that we need to dedicate significant funds, comparable to the ones that we had under EU for health in the previous budget, to health policy proper. And also, we need to dedicate a certain amount to competitiveness in the narrow sense, which means industrial policy in the field of health, pharma, etcetera.
We also need very clearly to dedicate funds to preparedness, military health, and digital health. And once again, here, we see a broad agreement between the different groups that such funds need to be dedicated. So I don't expect to have any conflict with colleagues. There is one significant difference, and I would say maybe only one significant difference, between the different groups, and it is whether health funds should be earmarked within the window as proposed by the commission or ring-fenced outside it, in a separate window. I must underline that we have more or less an agreement about the amount that we need to ring fence, but not whether it should be in a separate ECF window or within the one proposed by the commission.
In my view, it is more of a symbolic question. So, I hope we will reach agreement here, because what is really important is to maintain health policy as a European priority and to make sure that we have enough funds for that. Thank you.
Speaker 27
Thank you, chair. Let me start by thanking the rapporteur for his work and for the constructive approach taken so far. From the S and D perspective, our amendments focus on ensuring that health policy retains its integrity within the future framework. In particular, we propose to separate health from the current health, biotech and bioeconomy window and establish a distinct health window with its own objectives and governance. This is reflected consistently across our amendments, where health is treated as a standalone policy area with specific objectives covering prevention, resilience of health systems, and preparedness for cross-border threats.
Speaker 26
On the budgetary side, we propose a dedicated allocation of €10,000,000,000 for health, ring fenced within the instrument alongside targeted earmarking for digital health and health preparedness in other windows. We also insist on maintaining continuity with the EU for health by removing its repeal and ensuring that its core actions are preserved in a separate health chapter. Finally, our amendments strengthen key horizontal elements, equitable access to health technologies, support for patient organizations, and the inclusion of health considerations in areas such as digital and defense, including medical countermeasures. Our objective is not to weaken the overall instrument, but to ensure that health investments remain visible, predictable, and effective in the next MFF. We remain ready to work with Rappoteau and the other groups in order to achieve a good outcome for the European citizens.
And I believe that our common goal will be to defend our proposal in the Itra report afterwards. Thank.
Speaker 20
Thank you very much, chair. We thank all of the work done by the SANTE committee to introduce the health aspect, specifically on the European competitiveness fund. We're not really completely clear how this will happen in practice and have this health dimension, because this is a challenge. This will be based on specific resources, and Europe needs to get back its leadership position. Today, we are leaders in research, but we also need to be able to bring innovation to work in practice, and that this happens on European soil.
We need to strengthen our strategic autonomy and ensure that European citizens continue to be able to have the highest standards of health in the various centers. Talent is essential. Europe cannot continue losing talent. We need to make sure that companies, researchers see Europe as a place to develop and stay in. We also see that the fund is very focused on climate, security, and defense.
They are important, undoubtedly, but we shouldn't ignore the potential that health represents. We've got the Green Deal. This is one of the reasons that has made us lose competitiveness. If we want to be able to move forward, we need to make sure that we deal with this issue. We need to be aligned with the Critical Medicines Act, and make sure that the products really get to the patients.
All of this needs to be in line with national competencies and the specificities of each country. We need to be transparent. We need to make sure that there is accountability and neutrality. This should be the case for all of the funds, and it needs to be in line with technology because at the end of the day, we're talking about improving the health of European citizens, and this means that we need results. Thank you.
Vlad Vasile-Voiculescu
Thank you. I will speak on behalf of my colleague, Stine Bose, who was the shadow rapporteur on this file. So Renew Europe has put forward ambitious proposals, given that this is a decisive moment for Europe, building on the good text by the rapporteur. We are asking for €10,000,000,000 for a robust EU health budget as a clear signal to the call legislators. This is about protecting public goods that help both people and our economy.
Health is the foundation of our sovereignty and strategic autonomy, proposing a package of measures on biodefense, including vaccines, antimicrobials and dual use products. We are, our civil and military preparedness requires EU manufacturing, strong supply chains, and a strong DG era. It is much better and cheaper to prevent these capabilities from leaving Europe than to pay for them to come back. Furthermore, health and biodefense are enablers of the EU military readiness. Therefore, funds for this must be made available under the resilience and security policy window.
Thematically, we are proposing AMR, rare diseases, and women's health as the key priorities for the health window. Throughout the texts, we are emphasizing gender equality, global health, the link between health and climate change, and the importance of enabling the activities of health NGOs. Finally, we want to put in place horizontal enablers that will help Europe lead in biotechnology, including by stressing the role of venture capital and helping innovative companies to move from the lab to commercialization. With technical meetings ongoing this week, I'm delighted that we, as a committee, are already moving in the right direction. Thank you.
Speaker 31
Thank you. And, I would like to state our points on behalf of Majdulin Spai, who's our member responsible for the file. Dear president, dear rapporteur, dear colleagues, first of all, thank you for the work on this file. I think we share a common objective to ensure that health is not sidelined and remains a core priority within the ECF. This is precisely why we believe a dedicated policy window for health is essential.
Speaker 30
Health technologies and systems cannot be treated in the same way as agriculture or biotech. Without a clear and distinct framework, there is a real risk that health priorities will be diluted. From a green perspective, we want to emphasize that the development of health technologies must be public-interest driven. Innovation should not be guided primarily by profitability, but by public health needs. This means ensuring that new technologies are affordable, accessible, and equitably distributed across all member states.
Public funding must come with clear guarantees. We cannot support a model where public investments derisk innovation, while access and pricing remain driven by private interests. We also tabled amendments to ensure that support to civil society organizations in the health sector remains effective, as we know they face budget cuts often. Competitiveness must be understood more broadly. Reducing pollution is not a constraint.
It is a driver of competitiveness and resilience. Preventing disease lowers health care costs, strengthens our workforce, and improves economic sustainability. In this context, we must take seriously the health impacts of harmful substances such as PFAS and endocrine disruptors. Addressing these challenges is not only environmentally necessary, but also a key component of a forward-looking and competitive European health strategy. Thank you.
Thank you.
Catarina Martins
Us on the left, clearly, we see the budget as being at risk. There'll be less budget for areas other than defense, and we're going to see cuts, clearly. And at a point in time where health is losing its own funding and is going to be folded into this fund, there could be less money for it. That's why we've tabled our amendments. Thank you to the rapporteur.
But our amendments are to guarantee health has its own funding, and that the program CEU has developed can continue and deliver for Europeans as a priority. Rare diseases, critical medicines, Alzheimer's, and obviously, women's rights to get care and to have an abortion. Then there are amendments calling attention to the whole question of principles. The commission has been in force for a year now. This budget is one of the first tests of whether the commission can ensure that public health prevails throughout the EU.
Or, in fact, is the commission there for the opposite purpose? We've got a broad majority from almost all groups that are writing a letter to the Commission. We started this when we realized what the budget would imply, saying that we need €10,000,000 at European level to fund programs we've been developing. My appeal, therefore, to you would be that when we compromise on the amendments for this opinion, that we shouldn't, in fact, lose sight of our original goal, a guarantee that what we're defending goes through, because European democracy can't make promises that are not funded properly. Thank you.
Adam Jarubas
Christine Anderson, ESN, please. No? Okay. So we have an indication from Mr. Claudio, please.
Christophe Clergeau
Yes, I would like to underline, to insist on three elements Christian Wieggenin developed. The first one is that we all agree on the amount of money we need. There is debate how to set it, how to implement it. So we are open to any scenario in terms of within or out of ECF. But if it's in, we need a regulation to implement it.
If we would implement it only through ECF regulation, there would be a high level of risk not to be able to really address the public health issues. So our idea is to delete the deletion of the current EU health legislation up to the commission to update it later. But we absolutely need legislation or regulation. It's totally key. The second element is that we need health in all the windows of ECF, including defense, security, and resilience.
And the third one is that we need health in Horizon Europe. And then we have difficulty because the report of the opinion is not about Horizon Europe, it's about the ECF. So we also have to take into account what's happening in ITRA committee about Horizon Europe. For example, when Christian Heller proposed to delete the missions in Horizon Europe, he proposed to delete the Conservation. So do we accept as some committee the end of a conservation in 2030?
Or shall we set it in the ECF? So we have to address this question, and we are a set of MEPs also to advocate for an exposome mission. So we have to define this capacity to mobilize money for research and innovation in link with a very important collective issue at European level. Concert is one, exposome is one over. So I think you will have as for important shadows also to address precise leveling between ECF and Horizon Europe, even if the opinion is not about Horizon Europe.
Adam Jarubas
Thank you, Christophe. Is there anyone who wants to take the floor now? If not, we are going to the commission representative, Missus Tatiana Lopez Garido, deputy head of the public investment unit in DG Grow, for her reaction for two minutes, please.
Speaker 33
Thank you. I would like to start to thank the subcommittee, of course, and especially the rapporteur for the draft amendments to the SAF proposal and also to the shadow rapporteurs. The commission has examined it carefully, and we have also listened to all your interventions today. We do take note of them. I would like to highlight some elements.
The commission's proposal for the new MFF demonstrates that indeed investigating population health and a strong health system is the foundation for social resilience, economic growth, and competitiveness. This is why the proposed ECF, together with Horizon Europe, union civil protection mechanism on health emergency preparedness, cover all thematic priorities of EU for health. The ECF will support actions to improve public health, strengthen health systems, and enhance population health outcomes in the EU, which will, in turn, improve productivity and competitiveness in our economies. This is firmly embedded in the proposed ETF regulation. It will deliver on core policies.
Those are health security, Europe's beating cancer plan, safe hearts plan, the European health data space, among others. In addition, the ACF will support medical innovation as well as strategic autonomy in medicines, medical devices, and medical countermeasures in line with the critical medicines and biotech acts and the medical countermeasures strategy. Just to conclude, with a tight connection to Horizon Europe, the ECF will deliver on the seamless investment journey, ensuring that European industry leverages union-funded research results to further innovate and produce in Europe. Thank you very much.
Adam Jarubas
Thank you. Finally, I give back the floor to Mister Kaneff for his final comments, please.
Speaker 24
Thank you, chair. As we all witnessed, there is really a very strong spirit of cooperation between the shadow rapporteurs, and I hope all the MEPs engaged. I must say, here first, I fully understand the reasons, mainly of our s and d colleagues, for a very clear ring fencing of health, health budgets and also earmarking of health funds in other policy windows under the ECF, and I'm sure we will find a solution together here, because we look in the same direction. And then, just to underline that one of my priorities as the Santa Proteur is to stay as strictly as possible within some competencies, which, of course, we might have different understanding on where exactly they stand. But, I think that any overstepping with the competencies of either Itra or the budget committee here might only be counterproductive.
I am in very, let's say, cooperative relation with ITRE colleagues, and I think we must also cooperate with Budge as much as possible. For example, I'm very much interested in the whole exposome concept proposed within the opinion by the S and D colleagues. But as far as I understand it, I think it very much belongs to the Horizon file and not to the ECF. So I would be very helpful as much as I can to have it included there, but I'm not sure it really belongs in the ECF regulation as a whole and in the Sandd opinion specifically.
Adam Jarubas
Thank you, Aradan, for your work. Dear colleagues, please note that opinion giving committees have been asked to deliver their opinion to Itra by 24th April. The vote on the Sandd opinion is currently scheduled for 14th April. Dear colleagues, our next point is the reporting back to the committee on the trial of the Critical Medicines Act. On March 16, the second trial of the CMA file took place at the council premises, during which several political issues were discussed between the legislators.
The meeting was conducted in a very good atmosphere. I would like to thank the rapporteur and shadow rapporteur for excellent cooperation. And now I will give the floor to Thomas Lav Sokol, our rapporteur on this file, to report back on the political discussion of the trial, please.
Tomislav Sokol
Thank you. Thank you, Chair, very much. As you said, on Monday we had the second trial. It was conducted in a good atmosphere, and we did make some progress. First, on the scope and definition of medicinal products of common interest, discussions led to encouraging but still limited progress.
The council demonstrated a degree of flexibility regarding the inclusion of orphan medicines within the definition of MPCI, as well as elements of the scope related to the permit granting procedure, pending further information to be provided by the commission. On the other hand, the parliament indicated some openness to narrowing the scope further, compared to its initial mandate. It is nevertheless important to note that the European Parliament remained united in its position throughout the discussions, and we have a very strong mandate here, which obliges us to stick to the most important priorities that we had relating to this. And I also think that before we get to the most contentious issues like joint procurement, redistribution, and European preference, the question of the scope and definitions needs to be solved. Second, concerning timelines, an agreement was reached on the objective of shortening the deadlines for procedures related to the determination of strategic projects and environmental assessments as much as possible.
The issue has been referred to the technical level with the mandate to further explore and identify a suitable and balanced solution. Third, regarding the strategic objectives of the union in article 4, a provisional agreement was achieved. The co-legislators agreed to retain the article, revise its title, include an explicit reference to patient safety, and maintain paragraphs two and three as proposed by the commission. More broadly, there was a shared understanding among the institutions of the importance of this act and the need to advance the work constructively.
Tomislav Sokol
Again. Okay. As usual. Okay. So more broadly, there was a shared understanding among the institutions of the importance of this act and the need to advance the work constructively and efficiently.
And if I might add, taking into account everything that's going on in the global context, especially in relationship with the US, but more general, this shows the importance of being less dependent on imports of medicines from third countries, both generic medicines and innovative ones. And in that sense, I think that what we have and what we propose in this legislation is of crucial importance to protect European industry, to maintain pharmaceutical industry in Europe, but also to achieve equality of access under the same conditions to medicines for all European citizens. Overall, while progress was made on several fronts, further technical work will be necessary to bridge remaining gaps, as I said, especially on the scope and the definitions, which I really think need to be resolved before we get to the most precious, most pressing, and most contentious issues in the end. So, thank you.
Adam Jarubas
Thank you, Tamislav. Let's move to the next point. Our next point, which is the proposals for pilot projects and preparatory actions for the 2027 Union budget for pre-assessment by the Commission. We have received 13 proposals for pilot projects in total that we will vote on today. The budget guidelines suggest voting on each of the individual proposals, and then a roll call vote on the package of proposals.
Yeah.
Five minutes.
Speaker 38
Five minutes break for our colleague to come to the room, please. Okay, colleagues. Let's get started. Please take your seat, and we will start in a moment. Now, we move to the next point, which is the proposals for pilot projects and preparatory actions for the 2027 Union budget for pre-assessment by the Commission.
Adam Jarubas
As I said, we have received 13 proposals for pilot projects in total that we will vote on today. The budget guidelines suggest voting on the repeat of the individual proposals and then roll call vote on the package of proposals. Let us now proceed to vote. First, testing vote. The question is, do you like spring?
I open the vote. I close the vote. Okay. Okay. As I said, we will be voting on each of the individual proposals by raising hand.
And after that, a roll call vote on the package. So let's start with the first proposal, European Rare Dice Lighthouses. Who is in favor? Thank you. Who is against?
Abstention adopted. Proposal two, strengthening EU collaboration on newborn screening for rare diseases. Who is in favor? Against. Abstention adopted.
Proposal three, closing the disability health data gap. European pilot for inclusive health data and AI. Who is in favor? Against abstention, adopted. Proposal four, accessible AI for inclusive health care.
Who's in favor? Against. Abstention adopted. Proposal five, women and cardiovascular health, improving awareness and early detection of heart attack and stroke symptoms. Who is in favor?
Against? Abstention. We should check electronically. I open the vote. I close the vote.
Adopted. Proposal six, connecting generations, weekly activities to improve well-being and reduce loneliness in elderly residents. Who is in favor? Against? Abstention.
Let's check, please. I open the vote. I close the vote. Adopted. Proposal seven.
The first thousand days building the biological, emotional, and cognitive foundation of life. Who is in favor? Against? Abstention ? Let's check it.
I open the vote, please. I close the vote. Adapted. Proposal eight, postpartum health and recovery monitoring, pelvic floor and abdominal health in women after childbirth. Who is in favor?
Against abstention. We should check it. I open the vote. I close the vote. Adapted.
Proposal nine, the case navigator. Who is in favor? Against. Abstention adopted. Proposal 10, saving organs in Europe, a pilot project to improve safe transport, monitoring, and recovery of liver, kidney, heart, and lung grafts through transportable machine perfusion.
Who is in favor? Against. Abstention adopted. Proposal 11, improving maternal nutrition through access to organic food during pregnancy. Who is in favor?
Against abstention adopted. Proposal 12, AI-based virtual patient simulation to improve early detection of chronic neurological disease progression in Europe. A pilot project evaluating digital training technologies for clinicians across the EU member states. Who is in favor? Against.
Abstention adopted. Proposal 13, diversity in patient representation within the ERNS. Who is in favor? Against abstention adopted. And as I said, now I open the roll-call vote.
I close the vote. Yes. We've got it. Thank you. Adopted.
Dear colleagues, our next two points are the votes to confirm the provisional agreement resulting from the inter-institutional negotiations on the two files of the pharma package. With these two votes, we will formally close the interinstitutional negotiations. And if adopted, I will send a confirmation letter to the chair of Correper to indicate our support for the agreement. As the parliament's first reading vote took place already in 2023, we are now in the early second-reading procedure. Thus, the procedure before parliaments adopts the legislation in plenary is different and a bit longer than for an early first-reading agreement.
In the coming weeks, the council will prepare the translation and the legal linguistic finalization of the two files. When this procedure is concluded, council will formally adopt its first reading position and will transmit it to the parliament. I have been informed that council expects to adopt its first reading in the second half of September. After an announcement of council's first reading position in plenary, possibly in October 1 session, the file will return to us the first session. The file will return to us as Sand Committee. We as Sandd will then vote on the draft second reading recommendation proposing to approve the Council position at first reading.
After the Sandd vote, plenary will decide whether to adopt the pharma package in the early second reading. We would expect this final vote to happen at the earliest during October, the second plenary session. Right now, I will give the floor to the reporters of both files, Timo Volcan for the regulation and Dolores Mosserat for the directive. After their interventions, we will have two separate roll-call votes, one for the regulation and one for the directive. In these votes, you will decide about your approval of the results of the inter-institutional negotiations as provisionally approved by Coreper in March.
Due to the strike in Germany, Timo has not managed to arrive at our meeting, and he will connect online. Timo, the floor is yours for 4 minutes, please.
Tiemo Wölken
Yep. Thank you very much, Adam, dear chair, dear colleagues. Indeed, my apologies for not being there today. We have workers' human strike in Germany, and my flight got canceled. So, but while I'm at it, I would like, obviously, to express my solidarity with all colleagues fighting for better working conditions.
So, very quickly, since we finished political discussions on this in December, the technical team has continued its work, and it has been very busy to close more than 400 articles of the pharma package. And I'm very happy that we can vote on the provisional agreement today. Or you can, I can't, unfortunately, but I would invite you to support it. The deal marks a major step towards a fairer, more accessible health care system for all EU citizens and a stronger, modern pharmaceutical framework. In a nutshell, we can, we came one step closer to a true European Health Union.
The regulation includes two new incentives for priority antimicrobials, placing the fight against AMR at the forefront, and this was our priority. By introducing a subscription model as a tool, we aim to further fix market failures by delinking company revenues from sales volumes of antibiotics, whilst encouraging member states to collaborate and to make the novel product throughout the union available. Transferable data exclusivity vouchers on top will help drive research and innovation for our priority antimicrobials by giving the right to twelve additional months of data protection for one authorized product. So the voucher is worldwide, one of its kind. So we're at the forefront, and I think as a union, we are showing leadership by adopting this incentive.
We furthermore optimized the functioning of EMA. We reduced the duration for the committee for medicinal products for human use, issuing a scientific opinion on the medicines marketing authorization to 180 days. We simplified the functioning of EMA's internal work and enabled the agency to treat market authorization requests more rapidly, allow the submission of electronic authorization applications, and eliminated unnecessary administrative burden but without jeopardizing safety for patients. Furthermore, we secured equal participation of patients and health care professionals, including the voting rights in the committees. And their involvement ensures that the regulation of medicines in Europe remains scientifically based, but also socially legitimate and firmly grounded in real world experience of the people where we are happy to see, helping us when we are in need. So when it comes to often additional products, we also introduced the category of breakthrough products, incentivizing research and development on products that tackle orphan diseases for which no treatment exists.
While the duration of exclusivity is now nine years for an orphan product, breakthrough receive an additional two years, so we are sending a clear and real political signal. We take this seriously, and we fought for the patients affected by rare diseases for which no treatment unfortunately exists until now. So the regulation will, furthermore, lessen, you know, shortages by requiring companies to put in place and update shortages prevention plans, and we continue this work with the Critical Medicines Act where Thomas Laff already gave an update on the last trial. All in all, I would like to thank all technical teams from all institutions that have been working so hard on finalizing the text after we received the political agreement back in December. I would also like to take this opportunity to thank again the Danish and Cypriot presidency for their commitment to fight a compromise and, obviously, to the whole parliament's team and also to you, chair, for helping us facilitate the compromises.
As of now, the text will be further looked at by our lawyers, as you already explained, Adam, and finalized so that we can have the final vote in council and parliament after the summer. And I would like to underline once again that I would be very happy to see the broadest possible support of this package because it really matters to the people in our union. Thank you very much. And once again, sorry for not being there.
Speaker 41
Yes. Thank you so much. Well, this directive, we cannot understand without the regulation because both are the big package. So I want to thank Timo and all the shadow rapporteurs in Timo's team. Because I think that today, for all of us, it is a privilege because we succeed in delivering the most ambitious legislative reform the European Union has ever undertaken, putting patients in the center of our European pharmaceutical law.
The first thing that I want is to thank all the shadow rapporteurs in our team in the directive and, of course, the committee chair, Adam, the secretariat and all the teams of advisers of all of us, all the groups, and of course, my own team, without whom we would not have been able to shift this historic result. We have to think that the trilogue agreement was reached in just four months of effective negotiations, a short time frame, especially considering that there were two legislative texts comprising over 400 articles. It is true that this work began much earlier in the last term. But after 20 years, it was mandatory to modernize pharmaceutical legislation focusing on four objectives. The first, to secure more and better treatments for all Europeans.
This is the first objective that we deliver. Second, ensuring the competitiveness of the European sector. Third, pursuing the economic sustainability of our national health systems that are universal in all Europe, in all our member states. And fourth, tackling current and future threats and medicines shortage, such as antimicrobial resistance, that it's a silent pandemic or this medicine shortage and increasingly serious problems in Europe. The most important results that we have achieved are as follows.
First, this legislation ensures investment in research and innovation for the best treatments, particularly to develop medicines for diseases that currently have no treatment, such as rare diseases and pediatric cancers, with a total data and market protection until 11 years, with an additional year in the case of adding antimicrobial voucher. Not only have we protected the intellectual property and market presence of innovators, we have also ensured that generics and biosimilars can be available from the very first day the market protection expires. So the day after the market protection expires, we will have biosimilars and generics. So this is the reason that we put the BOLAR clause. Third, I would like to highlight the response to what is now one of the main threats to public health: antimicrobial resistance.
I have to say to you that it has been my obsession. It's amazing. You know? Every year, 40,000 Europeans are dying from antimicrobial resistance. And for me, it is a silent pandemic.
And we have to focus on that to put solutions. So there will be a mandatory prescription requirement for all antibiotics, harmonized in all member states, along with a warning label regarding their misuse and the possibility of further restrictions in the event of a threat of resistance. So when we are going to buy an antibiotic with a prescription because it has to be obliged this prescription in the pharmacies, in the box of the drug, you will see inside a red card saying this is antibiotic. Be careful. It has to be on the prescription.
If you have some doubt, go to your doctor or pharmacist, and you have to follow all the treatment. Another point I wish to highlight is that this package recognizes the vital role of pharmacists, which are the first point of access citizens have to the health care systems in any town, even the most remote. Because Europe is so big, we have 27 countries, and we have a lot of rural zones where the first health care point is the pharmacist office. So we wanted to give this importance to them. And we have retained the provision allowing member states to authorize pharmacies to prepare compounded medicines for a limited period in justified circumstance for patients for whom no authorized medicine is available, provided there are prescribed by a doctor.
So I think that this point is very important for, you know, those patients that they are chronic disease, and we have to adapt sometimes the medicines for them. So this point is very important. And then I will also like to highlight that in Petus, we have given the digital package leaflet. Member states will be able to choose whether to have only a version or both the digital and printed version. The digital format, which could be accessed via a QR code, for example, makes information on the medicine more accessible to everyone.
I'm thinking for the people that they cannot read these small letters. So, you know, for some disabilities, it's going to be very good. And, of course, all the languages. Imagine that I'm a patient from Spain. I'm going to Greece.
Always, I'm explaining this case. I'm going to Greece. I'm in the hospital, and they are Cyprus. And they prescribe me a medicine. I'm going to the medicine office, a pharmaceutical office, and I don't understand the leaflet because it's in Greek.
And for me, then in the Google or artificial intelligence, it's so complicated to put the leaflet in Greek because it's another no. It's not the, it's a Greek word. It's not a Latin word. So in the QR, we are going to have the 24 languages too, about that medicine. Last but not least, I would like to highlight that a new article has been added to facilitate access for member states requesting access to medicines.
We don't want to Europe, the first Europe and the second Europe, the one that they have access to the medicines and the second one that it's so complicated for them, the patients to access the medicine. So the member state may request procedures towards companies to ensure access. If three years after the request, the product is not being supplied in that member state, the company, the industry may lose market protection in that member state, but only in that member state. This will facilitate access to medicines in that country, whilst maintaining the overall incentives. And thank you very much again to all of you and all the stakeholders, especially the patients who guide us to reach the most ambitious and adequate European pharmaceutical legislation reform of the last decade.
And I hope so that not just this Sante Committee, all the plenary support in this important package. So thank you to both teams because it was one of the first times that two teams trying to bring to you this important pharmaceutical package. Thank you so much, President.
Adam Jarubas
Thank you so much, Dolores. Once again, many thanks to our reporters, secretariat, and advisers. It was really great work on difficult files. Dear colleagues, let's now proceed to the vote. As I said, first roll call vote for the regulation.
I open the vote. I close the vote. We've got it adopted. And the second one, roll call vote for the directive. I open the vote.
I close the vote. Adapted. The Senate Committee has hereby approved the outcome of the inter-institutional negotiation on the pharma package. Congratulations. Dear colleagues, now I would like to ask my colleague, vice president Tilly Metz, to replace me for the ninth point of the agenda.
One moment.
Speaker 43
One moment before we start. Yep. Two minutes. Okay. I think we should start.
Tilly Metz
Please take a seat. Happy to have this workshop on rare diseases. So dear colleague, our last point this morning is the workshop on rare diseases. This workshop has been organized by the policy department to support our work on the legislative own-initiative report on the EU rare disease action plan. Policy department has invited three experts, Doctor.
Joak Wietz, I hope I say it right, Weitz, who is a team leader on rare disease in Cienzano. And then we have Doctor. Annabel Callum, who is the national coordinator of Orphanet Belgium. And then online also, because of the strikes, we have professor Annette Grutos Kilis, who is a physician at the Charité in Berlin, so the university medical center. So first, doctor Witz and doctor Kalom will make a presentation from data to diagnosis, building a European rare disease data ecosystem.
And then as a second, Professor Agutis Kilis will make the presentation on challenges and opportunities for health care and research in rare diseases in the AU. And after the two presentations, I will give the floor, of course, to Nicolas Gonzalez Casares, who is the S&D rapporteur for this file, and then to our shadow rapporteurs also. So, after their interventions, I will also give the invited experts a possibility to react for comments and questions. And all other members here will have the possibility to take the floor in the second round of intervention. As usual, our discussion will be based then on Catch DI.
And I invite members who want to take the floor to indicate it to me and the Secretariat. So now, with no further delay, I give the floor to the first speaker, Doctor. Wits, doctor. Calme. The floor is yours for 10 minutes in total.
Please, of course, try to restrict the timing. Thank you very.
Speaker 45
Much. Thank you. Dear members of the European Parliament, dear Madame Dirsir, good morning, and thank you for the invitation. So, as you may know, rare diseases are individually rare, but collectively common. They affect more than 30 million people in the European Union.
Yet one of the biggest challenges these patients face is not only medical. It's a data problem. Rare disease data are scarce, and they are difficult to connect across countries. This makes diagnosis slower, research more complex, and policymaking less effective than it should be. Today, we would like to show you how better connecting rare disease data in Europe, in full compliance with EU data protection rules, can help move from fragmented data to faster diagnosis and also better care.
Because patient populations are small and geographically dispersed, the available data are spread across hospitals, registries, and countries, often using different standards and coding systems. As a result, their value remains underused. This is why improving data pooling and interoperability is essential. Enhancing the data collection and analysis, but also ensuring that these data are fair, which means findable, accessible, interoperable, and reusable. These are key to unlocking their full potential for research, for care, and also for policy making.
Next slide, please. One of.
the key.
Speaker 45
Infrastructures supporting the rare disease field in Europe is Orphanet. Orphanet is the main European reference knowledge base for rare diseases, providing reliable information on diseases, also on expenditures, and on all activities related to research. It also provides a structured nomenclature for rare diseases known as Orpha codes. Importantly, Orphanet is recognised within the framework of the European Health Data Space, highlighting its role in connecting rare disease knowledge, registries, and research across Europe. In this way, Orphanet forms one of the foundations of the European rare disease data ecosystem.
Next slide, please. One of the key challenges in rare diseases is that many of them remain invisible in health information systems. This is partly because many rare diseases are poorly represented in generic coding systems, such as ICD or SNOMED CT. In addition, different countries often use different terminologies, which makes it difficult to connect data sets across borders. As a result, rare disease data remain fragmented and difficult to analyze.
Orphacoats help address this gap by providing a dedicated terminology that enables the stricter recording of rare disease diagnoses in the health records. This supports both the secondary use of the data, such as for research, epidemiology or clinical trials recruitment, but also better patient care by improving the visibility of the diagnosis and facilitating the referral to expert centres such as ERNs. And also support access to appropriate treatments. Next slide, please. Another key pillar of the European rare disease landscape is the European Reference Networks.
They enable the experts to collaborate across borders, share knowledge, and discuss complex cases through virtual consultations. They also support collaborative research, including the development of international registries. However, significant data-related challenges remain. We can mention the development of the year-end registry. This is still really uneven across networks, with limited standardization of data elements, coding systems, and technical infrastructures.
In addition, many registries operate on a voluntary basis and remain underused due to limited incentives and a lack of visibility. Initiatives such as action aim to address these challenges by improving the interoperability and strengthening coordination across Europe. Next slide. Registries are, we know that, extremely valuable tools, particularly for rare diseases where patients' populations are very small. They support clinical research, and they generate real world evidence that helps policymakers to make evidence-based decisions.
However, their potential remains underused. Today, around 800 rare disease registries exist in Europe, but most of them are organized at the national level, which creates what we can call data silos. In addition, differences in standards, but also in consent models, can complicate cross-border data sharing. Building a federated and interoperable European data structure aligned with the European Health Data Space would allow rare disease registries to fully realise their potential. Next slide.
Improving the diagnosis also requires better support for frontline clinicians. Most patients first enter the system through primary care. Yet rare diseases remain difficult to recognize, and patients often experience long diagnostic journeys before reaching the right specialist. Several actions can help address this, including targeted awareness campaigns, capacity building, and the development of digital health tools that support the general practitioners and clinicians in everyday practice. One example is the rare disease knowledge tool, which is co-developed by Orphanet and the Yerens.
This medical device helps healthcare professionals explore possible diagnosis hypotheses and identify the most relevant expert centres. By making specialised knowledge more accessible, these kinds of tools can help shorten the diagnostic odc for patients with rare diseases.
Speaker 46
Next slide. Improving diagnosis and care in rare diseases requires a stronger connection between the specialized expertise on the one hand, and the frontline health care professionals on the other. While ERNs bring together highly specialized centers, most patients are first seen by primary care and second-line health care workers. Strengthening the collaboration between orphanage, ERNs, and clinicians would help ensure that expert knowledge, such as clinical pathways, guidelines, practical tools, reach those who see the patient first. This would make reliable information more accessible to clinicians, reduce duplication of work, and ultimately result in faster referrals, earlier diagnosis, and overall better care pathways for patients.
Speaker 47
Next slide. This slide essentially summarizes our main message: Improving outcomes for rare diseases requires a connected European rare disease data ecosystem. It starts with making rare diseases visible in the health system through ORPHA codes and common minimal data sets. These are sets of data elements, for example, clinical, diagnostic, demographic information that are collected in a standardised and structured way across countries, across registries, and health care systems, which allow the health data to become interoperable. Once data can be connected, they can support ERNs and registries, which help generate knowledge.
And this knowledge can then feed into research, innovation, digital tools, and ultimately help clinicians reach faster diagnosis and provide better care. On the next slide, yes. This is actually a concrete example of what an ideal data ecosystem for rare diseases could look like in practice. On the left, you see the primary use of data, where the goal is to ensure the best possible care for the patients. Once a patient is diagnosed with a rare disease in an ERN center, for example, their medical records should include a structured data set describing their specific diagnosis, thanks to an ORPHAcode.
Speaker 46
These data should follow the patient throughout its journey during rehospitalizations, emergency visits, but actually with every contact with another healthcare professional, and also when traveling to another country. At the same time, this data can also be very valuable for secondary purposes, provided they're properly structured, of course, and once they're pseudonymized. By combining rare disease data with other demographic data and health data in registries, we can better understand the health care needs, the societal and economic impact and burden of rare diseases, and support evidence-based policy making. Aggregating these scarce data at the European level would help reach the critical mass needed to accelerate research, diagnosis, and innovation in rare diseases. On the next slide, we've listed many initiatives that are already in place and work towards a European rare disease data ecosystem.
Speaker 47
By collaborating with clinicians and researchers, they're testing solutions, providing practical tools, sharing best practices, and making recommendations to improve interoperability. Building on these existing resources is essential. And then finally, to conclude, yes. To conclude, rare diseases are one of the clearest examples where European collaboration creates real added value. No single country has enough patients, enough expertise, or data to address the thousands of rare diseases alone.
This is why coordinated action at the European level is essential. In practice, in practical terms, this means promoting the systematic use of common standards, strengthening interoperability between registries, ensuring that the expertise developed within the European reference networks reaches the patients, and continuing to invest in tools, digital diagnostic tools, but also investing in resources more in general. These priorities are fully aligned with the vision of the European health data space, which helps transform fragmented rare disease data into faster diagnosis and better care for millions of rare disease patients. And with that, we thank you for your attention.
Tilly Metz
Thank you very much to you. And you nearly stick to the time. That's perfect. So again, due to the strike in Germany, professor Grutus Kielich has not managed to arrive here in Brussels for a meeting, and she will connect online. So, professor Grutus Kielich, the floor is yours also for 10 minutes.
There you are. Welcome. I'm a good we will check if the sound is good, if we can I don't know in which language you wanted to speak?
Will present in English.
Okay.
Can you hear me? Can you hear me well?
Yeah. That's fine. That's fine.
Okay. And then, okay.
Tilly Metz
Yeah. I hope it works for the interpretation in other languages then, but let's see. I'm looking for a sign. No. Okay.
Speaker 48
But then you speak in English, then all is good. I would, yes, okay. Thank you for being there.
Speaker 49
So can I have the first slide, please? So thank you very much for inviting me to give a short impart on the challenges and opportunities for healthcare and research within the EU. Myself, I am a pediatrician and also a clinician scientist, and I am also the Vice President of Arlea of All European Academies of Sciences and Humanities in Europe. The next slide, please. As you heard, rare is many, but in fact the unmet medical needs of patients with rare diseases are really neglected in many also of the European countries and regarded as of minor interest.
I prepared this slide to give you probably some new perspective on the numbers, because we know that 36 to 40 million people within the EU are affected with a rare disease. This is a similar number to the number of patients who were treated because of lipid disorders with statins. The majority of these patients are old, between 40 and 75 years, while the patients affected with rare diseases predominantly are children. In contrast to the patients with statins that are available to almost 100 percent within the EU countries, for the patients affected with rare diseases that account to 6,000 different ones, only 5 percent have an effective and targeted treatment. But probably the most important number on that slide is the 120 million EU citizens that are affected directly or indirectly.
Because most of the patients are children, there is a significant impact on the family, so at least on the two parents. Mostly the mothers reduce their ability to work, even skip their work and careers, and have a high burden of taking care. This is why rare are many, and not a minor entity. So the next slide, please. The next slide, please.
So another important number is that when you look at the whole number of rare diseases, 5% of all newborns are affected by rare diseases. And three of them are so severely affected that they won't reach their fifth birthday. And again, maybe an interesting comparison. So in the EU region, approximately 15,000 children and adolescents died of car accidents in the year 1978. And this number was reduced to 400 by the regulations that were put in place by the EU, especially the buckling up regulation.
For the rare diseases, a similar number of children and adolescents die of rare diseases, but there is no therapy available for most of the cases. Although with enforced research and innovation, there could be treatment, and there could be at least a cure in some of them. The next slide. This summarizes the challenges and opportunities, and the overarching issue for the EU is the inequities that we encounter in many respects. For the early diagnosis and access to specialized care, we have a mean duration of 5 to 7 years in the EU region, but there is even a larger difference when you go from country to country.
There is, of course, inequity in the access to therapies. I just was already present when the pharma package was voted on, and of course that will hopefully help, but there is a lack of interest in pharma in R&D for rare diseases, and this will change probably also with the pharma package most likely slowly. What's even worse is that we have a huge diversity in the reimbursement and prices of therapies for rare diseases in the EU region. But there are opportunities. I think that the case of rare diseases is something that really could have societal, I would call, side effects, that can show that there is inclusivity, mutual understanding and trust within the EU if we could reach for these small numbers of patients to a more common access to early diagnosis and therapy.
A first step was made by the creation of the ERNs, and also, as you heard in the previous talk, on having European registries for rare diseases. This will be important also to gain independence from non-EU countries in R and D. Also for research projects, I think there is a huge potential, especially when we would be able to create a European newborn screening initiative. I will go a little bit into more detail to that in a minute. The next slide.
For the challenge of the diagnostic Odyssey, yes, we have the ERNs and unsolved medical cases that may be a rare disease are referred to case discussions within the ERNs. However, for many of them, then advanced diagnostics, including sequencing, whole genomic sequencing, but also other advanced diagnostics, are necessary. These are frequently not available in a country in the EU region. What we really need is to create a mechanism that these patients who are discussed in these case conferences and where there are proposals for further diagnostics, that this could be really done, and therefore funds would be necessary. The next slide.
Going to newborn screening, as you can see here, there is a growing landscape of pilot programs of expanded newborn screening which are based on genetic markers. The current programs are mainly based on biochemical markers, but for many diseases where a therapy is already available, they can only be detected on the basis of genetic markers. They can be detected before symptoms occur, and can be effectively treated. As you can see, there is a program in Belgium, a program in France, there is the Screen4Care EU funded program, but only with rather small numbers when you compare it to the rest of the world. This is something we have to consider really seriously, which European initiative could be done to have an EU harmonized program.
Here next you see the different numbers of diseases that are covered in the newborn screening programs, ranging from 950 in one US program down to 250. However, when you look at what is shared by all of these programs, it is only 53 diseases. These are the diseases for which treatment is available. The huge diversity of these numbers that are covered by the pilot programs is clearly dependent on the people or experts that have created these programs with their preferences, including a lot of diseases where there is no treatment or maybe an actionable approach. I think what we really need urgently is that we come to consensus within Europe for which diseases we would like to have harmonized EU programs.
The next slide. And for therapies, well, there was the vote now on the pharma package, so I can make this very short. As you can see, we have this huge diversity in refunding or reimbursements of therapies ranging from 90 percent in Germany down to 27 percent in Poland. So roughly only 50 percent of the patients with rare diseases can get a reimbursement of treatment within the EU. One example is the therapy for spinal muscular atrophy.
Being a paediatrician and having seen these children for decades, where we had no treatment and saw them die, and now they live and develop normally, it is almost unbearable to accept that there are children within the EU that cannot benefit from this treatment. The next slide. When you look at the orphan drugs that are now approved by EMA, it is roughly 160 therapies, but 70 percent of them are for what they call rare oncological diseases with specific molecular footprints of tumor. Only fifty-one of them are for these inherited genetic ultra rare diseases. Also the fear that our healthcare systems will be overrun by the cost of these therapies.
I think when you look at the figures, it is stable, the number of orphans that are approved by EMA. I think we need above the pharma package, we have to think about how to differentiate incentives for the research and development of therapies between oncological diseases and genetic diseases that mostly occur in childhood. Next slide. These are my conclusions. I think that the EINs can only be a first step towards cross-border healthcare of patients with rare diseases.
We need to find a mechanism to finance also genomic sequencing and advanced diagnostic technologies across borders. The bottleneck of genomic sequencing, for example, could be overcome by really having a mechanism to accredit EU centers of excellence that then would benefit from funds that pay for genomic sequences for other countries. We need common principles for the screening of newborns, and probably a European Board of Experts and experts that are independent would really be needed to establish and also monitor in a rolling manner the establishment of a harmonized European newborn screening program. And the last one, last slide, and for the therapies, I think we need especially for the ultra rare genetic diseases with so few numbers, very special regulatory pathways and incentives, because otherwise we cannot motivate Big Pharma to invest in these developments. We need also more transparency across Europe regarding access, but also pricing.
We need sustainable EU funding and payment models, including some risk sharing or outcome-based arrangements, to speed up the process of therapeutic development, which is possible now. We need for research and R and D and innovation ecosystem that really focuses on rare diseases within ERA, and this is really necessary to achieve independency from the US and also from China and Asia, because it is really a threat that most of the therapies that are available now are developed and produced by US pharma companies, and access to therapy may be endangered in the future. Thank you very much for having me here and giving this impulse, and maybe it was a little bit too emotional, but I come from a perspective of 40 years of clinical and also research practice in rare diseases. Thank you very much.
Tilly Metz
Thank you very much, Vindank, to you, Professor Grutis Kisles. Now we go, first of all, to the rapporteur, Nicolas Gonzalez Casares, who has the floor for two minutes to speak on the EU rare disease action plan and his expectations there. And then I give the floor, of course, to the shadow rapporteur.
Speaker 50
Thank you.
Very much, Chair. Thank you very much to the experts for their contributions, presentations, and for organizing this panel as well. Rare diseases affect millions of people, and many are children, and the diagnostic period is very important. We have a series of diagnostics, which can often last years. Treatments are not always advocates.
This means lots of years of suffering for the families as well. So we have a clear and ambitious goal. Reduce the average diagnostic time to a maximum of one year throughout the European Union. And this is absolutely necessary, clinically, ethically. I mean, we've seen the presentations.
We need to interconnect the data, the solutions. And by doing that, we could reach this goal. We have the European Reference Network. We have infrastructure for data and structures like Orphanet. And these could allow for better interconnection of the data and make sure that we have robust data that is interconnected.
So at an international level as well, we've got a growing consensus on the need to have diagnostics based on genomic data and shared clinical intelligence. Not all intelligence is artificial, but clinical intelligence is based on human assessment. We don't want to put all of our hopes on algorithms. We ourselves have algorithms, and we have science. What we need is to connect all of these pieces together in an effective manner.
This is also a question of health justice. We also need to make sure that there is accessibility. And then just a final comment on the final comments that we heard. And this has a lot to do with what we've discussed in this committee. If a country spends a lot on defense, on arms, and can only pay a small percentage of medicines for children, and then can pay a lot of the reimbursement for the products of rare diseases, then what is really the priority?
Is it reducing the defense spending or the reimbursement for rare diseases? I know this is not what we're talking about specifically, but I think we need to think about it.
Tilly Metz
Very good remarks. Thank you very much to Nicolas Gonzalez Casares, and I give now the floor to the shadow reporter. And first of all, from the EPP, Maria Elena Nevado Del Campo.
Elena Nevado del Campo
Thank you, President. Thank you for your presentation. I will speak in Spanish, too. First.
Of all, I wanted to thank you for your presentations. And also wanted to ask if you could share those presentations with us, because I think they could be very useful for us in our work, specifically helping us with fraud. I've got two questions. First, with this data ecosystem that you're putting forward, how long do you think we'll need to invest in order to set this up? Because time is of the essence for our patients.
And then number two, have you looked at or thought about the investment we would need to make to make sure that we have this plan, this rare disease plan, and to make it functional? And then the third question, would we be opting more for a rare disease plan? Or should we be looking at a regulation on rare diseases that would be implemented in the member states? And you also talked about the need to make rare diseases more visible. Now allow me to tell you about Hector.
Hector lives in the rural countryside of Spain. He suffers from a rare disease. There are no treatments. There are no diagnostics, and he doesn't have time. He needs to make sure that all of these rare diseases are visible.
Therefore, we are committing to make sure that the patients have a European plan on rare diseases that can detect these kinds of diseases early enough. And so I would ask you to help us convince the commission and the council, because we are already very much committed here in the European Parliament, to not turn a blind eye. This is a moral obligation. Thank you.
Tilly Metz
Thank you very much. And the next shadow reporter is from the PFE. It's Marie Luce Brazier Klein. You have the floor.
Marie-Luce Brasier-Clain
Thank you very much, ladies, for the presentation. Rare diseases, it's very difficult to get a diagnosis. And there are many obstacles because of the lack of specialists. There are long waiting times and the cost of treatments if they even exist. Making things that work in the laboratory, commercial, it's very expensive.
There's a negotiation system between the public authorities and laboratories when it comes to prices, and it's regulatory issues because of trials, which are not appropriate for the requirements of rare diseases. So my question is how to get rapid authorization for placing on the market and incentives for the development of treatments. If the market isn't profitable because of the low revenue, and if the therapeutic value of the treatment is not easily provable, all of that affects what happens to medications. Now these things do exist, but there are different industrial strategies and different member states. There are different ways of evaluating things.
Are different administrative systems, etc. Obviously, it also comes down to the rather opportunistic and mercantile money-making approach of laboratories. Now there's a European approach. That's going to be essential because the country on its own can only have a few patients affected by these rare diseases. But if you scale it up to the European level, then there'll be many more.
But your report has shown that three quarters of patients aren't even aware of actions at European level. And worse still, the reference network isn't known about by the specialists, this European corporation, which also has the degree of red tape, which requires authorizations, and is rather form filling in its approach. Now how do we, therefore, try to speed up procedures? That's the fundamental of it.
Tilly Metz
And the next speaker is from Renu, and it's Billy Canera. Please, you have the floor.
Speaker 53
Thank you very much, Madam Chair. Thank you for the presentation. Again, I'd appreciate if they could be circulated to the members, very informative. Just a few observations. I mean, if you were just to look at it purely from a harsh clinical cold perspective in terms of health policy planning into the future, the issue of newborn screening is something that I believe that we have to put a lot of emphasis on.
When we talk about lack of data, when we talk about delays in diagnosis, when we talk about failure to actually have a pathway for care for patients, obviously, the most immediate issue would be newborn screening, a broad based European wide screening system that could identify rare diseases at an early stage, primarily for the individual patient, but also from a public policy perspective in terms of planning into the future. I mean, I know we have European reference networks, but we still have a very disparate health system across Europe when it comes to catering for people with rare diseases. So I suppose the key question here is, is there capacity available to roll out a broadly based screening program for newborns that would identify, like, bearing in mind, it's like a postcode lottery where you're born at the moment as to whether or not you will be diagnosed. In some countries, testing for eight or nine, and in other countries, up to 50. So it really is very imbalanced.
And with that then, I mean, if we had a profile of the rare diseases and the patient profile, well, it clearly, you know, the pharmaceutical companies and others could get involved in the area of looking at innovation and, you know, developing treatments, I should say, for rare diseases, knowing that there is a pool of patients that will require these treatments into the future. So if maybe you could elaborate on that. Just the issue I mean, you know, we can all talk about the hypothetical, until such time as there's actually a proper reimbursement system in place to stimulate investment in innovation, research in rare diseases, and ultimately, the return that's required for that high risk in the innovation investment. Where do you see Europe in terms of reimbursement? And how you know, is there costs on us in terms of the potential pressure that would be put on health systems?
Bear in mind, health is still a national competency. Decisions we make in this room will impact taxpayers in countries as opposed to directly on the European taxpayer themselves. So if you could maybe just elaborate on those issues. Thank you very much.
Tilly Metz
Thank you very much for your intervention. And now I give the floor to the Greens. And as Nikolay Stefanuta was not able to be there, I give the floor to myself. So first of all, thank you very much for your presentations. Very interesting.
And first of all, maybe two questions to the Doctor. Wirtz and Doctor. Callum. You said, and for me, it's not 100% clear what you mean by that. So that rare diseases are visible in the health information systems.
What concretely do you mean by that? For me, it's not 100% clear that we need more awareness, etc., on rare disease. I totally agree, but they are not present in the information system is not 100% clear. And then, at the end, you spoke about policy priorities. And then you enumerated four of them.
So of course, common standards and interoperability, this is key, and then integration of the European reference network, and also investing in digital tools. So the last two are very clear to me. But I would like to ask you coming really from the ground, in order to have these common standards and this interoperability, maybe do you have concrete steps how we can achieve that progressively and having also the really the health care professionals on board there? And then I have another question to professor Grutus Kislisch, because we hear that so often, we need an innovation ecosystem in Europe in order to foster, to boost innovation, especially regarding rare diseases. And then I would be curious also there, what exactly do you mean by that?
So to have really this ecosystem, what would be the key, if I may say so, ingredients of this ecosystem? And then last remark I would like to make is also a call on the rapporteur and on the shadow rapporteur. I think it's key to have a holistic approach in this action plan. So to speak also about patients' rights and inclusion regarding education, but also regarding employment. So really, and also considering the informal caregivers that often play an important role in the rare disease environment.
And then speaking about the environment, and it's, again, maybe also a question to the expert, don't you think we definitely need to consider also the environment and the environmental determinants? So I'm speaking about toxic exposures and about the prevention dimension. Should we not consider much more also in the data this toxic exposure that can lead at the end also to rare diseases? Voila, sorry for all these questions, but I found, as I said, your presentation very interesting. Thank you.
And also, I think that's it for there is nobody from the left and also nobody from ESN. So I think that's yes. So I give the floor back to the experts in order to react to all these questions. Thank you.
Speaker 45
Yes, thank you for your question. So I start by replaying the question about what do we mean about the lack of visibility of rare disease data in the health information system. So actually, the generic classification is not designed to fully capture the big diversity, heterogeneity of rare diseases. And I mentioned the ORFA codes, is really the dedicated terminology to rare diseases that is really created to accurately and with a big granularity represent the rare diseases in the health electronic system. When we talk about interoperability, it means that the different terminologies can be mapped together, so it means that you can have corresponding tables between the different terminologies to find the equivalent.
So it makes interoperability possible, but we really support the use of ortho codes as the primary coding in the health electronic records. Why? First, because an ortho code is already a rarity flag. So it means that immediately, if you have an ortho code in a patient file, the clinician knows that in front of him or her, he has a rare disease patient. And it can be very important in some kind of emergency situation, for example, where you have to do anaesthesia to someone who has a rare disease.
At least immediately, you don't lose time, you know you have a rare disease in front of you, a patient, and you can immediately also find all the information related to this disease via the Orphanet website, etc. The problem for the moment is that the use of them remains largely voluntary. It means that they often complement other national coding systems, such as ICD OrthoVelocity, but they are not in the primary coding system. And the adoption is very variable between the countries and the institutions. So what we ask for is really to make the Orphaco choose systematic.
And for that, we really think that a clear implementation guidance, but also a legal framework, would be useful to help the adoption, the integration alongside other coding systems that are national and mandatory. If I talk for my country in Belgium, it's Nomad City, with the terminology supported by the Ministry of Health. So the ORFA code is only used as a kind of extra layer. So it means that it's for secondary purposes, like sending data to the registry, but you don't have the ORFA codes embedded in the patient file, which is really a big issue. And you need also technical support to integrate these ORFA codes at the hospital level, so at the local level in the different kinds of hospitals, and of course incentive trainings to help the clinician to use this quote.
Speaker 47
Yes. To respond to the question related to the investments needed, time is of the essence. We fully agree. I think there are a lot of quick wins already that we can make. For example, there is the joint action Jardin, which will make recommendations as early as next year.
There have been a lot of tools developed, common minimal data sets that have been elaborated. So there have been made agreements between clinicians on what information is essential for these rare disease patients. I think once these recommendations are there, the next step is really to ensure that they don't remain just recommendations, but that there is a policy in place to ensure that these tools are being used. I think we have a lot of examples of initiatives where tools are being developed, but we also need the clear policy message. This is a priority.
Investments are needed when you need to make choices, like it was said earlier. But I must admit, it's also a long-term investment, without a doubt. Countries work at different speeds, have different levels of maturity. From the Jardin questionnaire, we also learned that only 56% of the hospitals have fully of the ERN centers that have fully electronic or fully digitalized patient records. So there is still paper-based work.
So even if there are tools in development for unstructured data that are very valuable, we must also take into account the European landscape and make sure that all the countries also follow with these developments. In terms of time as well, I think the European health data space is very promising to us, where the implementation is in progress, but it will take many years. And it will also be a question to us, how will this, in fact, change the landscape? We're currently having an EHDS rare disease task force to see what are the particularities that should be taken into account for rare disease data, specifically for the implementation of EHDS? I'm thinking about the risk of re-identification, for example, in case of secondary use.
But also, how will this regulation change the rare disease community? In terms of the question related to the rare diseases plan, should it be a regulation or implemented nationally? I think we're very much in favor that there is one clear message at the European level, a regulation. But I think it's also an end to end story, a clear message, clear priorities on a European level with the regulation, but also national plans will be essential to, yes, to be tailored to the specific health care system and to ensure that the priorities are implemented. And then maybe one last element that I wanted to add regarding the trials and rare diseases.
There are, again, their particularities. We have small populations. So if treatments are developed, it's really important that once they come to market, that they're also continuously followed up. How are these patients doing, especially with these new innovative treatments, such as gene therapies? Again, real-world data is crucial there.
We are currently already having registries where, when new innovative treatments come to market, we follow those patients years after years, and we see how they progress. Does this disease recur, or are there really long-term benefits? I think it's really essential to not lose that as well because this real world data also provides part of part of the answer. Mhmm.
Tilly Metz
Thank you very much. And shortly, now we are a bit over time, but I would like to give the floor back also to professor Grutus Kislisch, maybe to react also, please, and especially maybe to my question. That would be nice. We cannot hear you. I whoops.
Okay.
Speaker 49
Okay. Well, the first point would be I'm addressing the topic of newborn screening again. And I think what you were asking about capacities, I think what would be the first step is that we would have kind of a European guideline or regulation on what the minimal requirements are for an expanded newborn screening program. Then one could think about scaling up, for example, sequencing in some European hubs where it would be possible for some countries to send the material of the newborns. The other question regarding what do you mean by the ecosystem?
I gave one example, if one would have European centers of excellence that combine healthcare with research, many questions that are still open today because of small numbers could be tackled much more easily if there were assigned centers in Europe to do this work. Regarding the environmental effects, yes, I think we will learn a lot of this in the future because beyond the genetics there are epigenetic effects. We have seen clusters of neurological diseases that were not inherited, and really clustered. When you look into the epigenetics, there were changes, and it is quite clear that some toxic environmental agents can produce these epigenetic effects and then mimic inherited genetic disorders. So this is something we also should put more emphasis on, I agree.
Those are my comments.
Tilly Metz
Thank you very much. Very clear, very good to the three of you. Thanks a lot for your interesting presentation, and we will work on that, of course. So I closed today's meeting with that. We will meet again tomorrow at 09:00 in the same room, and there we speak about the biotech act, and the commissioner of Baheli is going to be there also for an exchange.
Is that right? Yep. So be there. Don't miss it. And now I invite the coordinators to stay in the coordinators' room and to stay here, so that we can have in camera the coordinators' meeting.
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